A BREAKTHROUGH discovery using stem cell technology could see motor neurone disease sufferers undergo the first clinical trials of drugs to defeat the illness within five to 10 years, according to Scotland-led research.
Scientists at Edinburgh University have been able for the first time to model the disease in the laboratory using stem cells, potentially paving the way for human clinical trials within the decade.
Clinical neurologist Sidharthan Chandran said the discovery was "pretty exciting", adding: "If you are asking how long will it take for treatments that go to clinical trial, from a discovery like this, the time I talk about is five to 10 years."
Motor neurone disease is an incurable and debilitating disorder that tends to kill patients relatively soon after diagnosis.
It is caused by motor neurons – nerve cells located in the brain and spinal cord that send signals to other parts of the body – breaking down, leading the sufferer to lose control of their muscles and becoming unable to speak, eat or walk.
Research has shown that a protein called TDP-43 is linked to more than 90% of cases, while 10% of sufferers carry a faulty gene.
Scientists at Edinburgh used skin cells from this second group to create the "dodgy mutations" which could then be compared against the healthy cells. They found that the mutant cells "died preferentially and selectively".
Professor Chandran said: "The power of this is that you can generate huge numbers of these in a laboratory dish using the stem cell technique and screen drugs to see which might slow down the death of the motor nerves."
Dr Brian Dickie, director of research and development for the MND Association, said: "This advance is a significant milestone on the road to developing a laboratory model of MND that faithfully reflects the cellular events happening in the patient."
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